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Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy

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Oligonucleotide-Based Therapies

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2036))

Abstract

Today, there are emerging numbers of oligonucleotide therapies being approved by the governmental authorities. These types of therapies present a different mode of action when compared to the traditional small molecules, acting at the RNA level instead of the protein level. In drug development, drug potency is defined by the drug affinity to the target biomolecule (target engagement), together with the ability to initiate a response at the molecular, cellular, tissue, or system level (efficacy). In oligonucleotide therapies, affinity and efficacy can be both easily evaluated by gene expression analysis. Although more advanced techniques can be used, this chapter presents a protocol to evaluate splice switching oligonucleotide efficacy that can be easily applied in a molecular biology laboratory without the need of advanced equipment. It describes all steps from sample preparation to data analysis.

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Author information

Authors and Affiliations

  1. Clinical Pharmacology & Safety Sciences, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden

    Cristina S. J. Rocha

Authors
  1. Cristina S. J. Rocha
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Correspondence to Cristina S. J. Rocha .

Editor information

Editors and Affiliations

  1. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Olof Gissberg

  2. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Rula Zain

  3. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Karin E. Lundin

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Rocha, C.S.J. (2019). Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy. In: Gissberg, O., Zain, R., Lundin, K. (eds) Oligonucleotide-Based Therapies. Methods in Molecular Biology, vol 2036. Humana, New York, NY. https://doi.org/10.1007/978-1-4939-9670-4_4

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  • DOI: https://doi.org/10.1007/978-1-4939-9670-4_4

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-4939-9669-8

  • Online ISBN: 978-1-4939-9670-4

  • eBook Packages: Springer Protocols

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